Parkinson病における病態修飾療法の現在と未来

抄録

Parkinson disease (PD) is characterized by the appearance of intracellular neuronal inclusions (i.e., Lewy bodies, LB) composed mainly of misfolded α–synuclein (αS). Under physiological state, αS is a small protein with no specific higher–order structure ; however, it begins to aggregate and acquire toxicity by gene mutation/duplication and various physico–chemical stresses. Therefore, many disease–modifying therapies for LB diseases/synucleinopathies have focused on reducing or eliminating abnormally aggregated, noxious αS. In this paper, I will first focus on the αS antibody therapies, which have attracted particular attention among disease–modifying therapies in synucleinopathy, and then discuss about the importance of patient stratification, which is a key for the success of clinical trials.